Biodegradable Highly Branched Poly(β-Amino Ester)s for Targeted Cancer Cell Gene Transfection.

To enhance the gene transfection efficiency to targeted cells while reducing the side effects to untargeted cells is of great significance for clinical gene therapy. Here, biodegradable highly branched poly(β-amino ester)s (HPAESS) are synthesized and functionalized with folate (HPAESS-FA) and lactobionic acid (HPAESS-Lac) for targeted cancer cell gene transfection. Results show that because of the triggered degradability of the vector and enhanced receptor-mediated cellular uptake of polyplexes, the HPAESS-FA and HPAESS-Lac exhibit superior gene transfection capability in specific cancer cells with negligible cytotoxicity, pointing to their promise as targeted vectors for efficient cancer gene therapy.